Advancing a New Patient-Derived Cellular Therapy for MS into the Clinic

Currently, there are limited treatment options for people with severe autoimmune diseases like MS, especially those who do not respond to existing therapies. Many people with MS experience ongoing disease progression and accumulating physical and cognitive disabilities despite receiving the most potent treatments available. MS primarily affects young adults, leading to significant societal and economic costs. Blood stem cell (SC) transplantation offers a promising approach by reducing inflammation and potentially providing long-lasting remission. Since an individual’s own stem cells are used, health risks are lower, but significant challenges related to safety and accessibility remain.

Research suggests that T-regulatory cells (Tregs), a type of anti-inflammatory cell, play a key role in the success of blood SC transplantation for MS. Dr Melissa Khoo and her team will investigate how Tregs stabilise MS by examining their gene and protein levels and their ability to reduce inflammation within the transplanted cells. Additionally, the team is working to produce Tregs on a larger scale to make SC transplantation safer and potentially reduce the need for high-dose chemotherapy.

Overall, Dr Khoo’s research aims to develop better treatment options for people with MS that match the effectiveness of SC transplantation while improving safety, accessibility, and quality of life. This work could have broader applications, offering new treatment possibilities for other autoimmune and non-autoimmune conditions, with the potential for wide-reaching impacts on clinical practice.